The Patient Voice: Is Anyone Listening?

Course Director: Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Sunday, February 4, 2024
4:00 – 5:45 PM

WORLDSymposium is presenting an exciting new program to be held on Sunday, February 4, 2024 from 4:00 – 5:45 PM just prior to the 20th Annual WORLDSymposium opening event, “Be the Catalyst“. This new session: The Patient Voice: Is Anyone Listening? will explore measures that industry and other researchers have taken to better understand what is most important to patients in terms of clinical trial endpoints, unmet needs, and goals of therapies. 

The goal of this new 1.75-hour non-CE session is to enhance awareness of the importance of listening to what therapy outcomes and unmet needs are most important to patients and their families, and incorporating those outcomes into clinical trial design and post-trial marketing follow-up. This session will be recorded and offered as part of the On Demand program through March 14, 2024.

Preliminary Agenda

4:00 PMWelcome (Jeanine R. Jarnes)
4:01 PMIntroductory Remarks from Family Perspective (Alan Finglas)
4:05 PMEarlier is better: Updates on a Phase I clinical trial of in utero enzyme replacement therapy (Tippi MacKenzie)
4:15 PMFrom Commitment to Co-Creation: Over 30 Years of Partnership with the Patient Community (Danielle Dong)
4:25 PMAs Unique as You: A Personalized Approach to Supporting the Patient’s Journey (Heather Park)
4:35 PMThe voice of caregiver study in MPS II and other notable caregiver observations made in JCR’s clinical MPS programs (Sairei So)
4:45 PMIncorporating the patient/caregiver voice in rare disease drug development using the biopsychosocial model (Heather Lau)
4:55 PMBest practices in patient education: How to create tools that meet community needs (Nita Patel)
5:05 PMEmbracing the silence: Taking listening to a new level. Mindfully rare mental health in the Fabry community. (Andres Trevino)
5:15 PMImproving Therapeutic Options in Cystinosis Through Partnerships with Patients, Caregivers and Healthcare Providers (Andrea Atherton)
5:25 PMClosing Remarks from Family Perspective (Cara O’Neill)
5:35 PMQ&A and Panel Discussion: Moderated by Matthew Ellinwood
5:45 PMAdjourn

Program Chair:
Jeanine R. Jarnes, PharmD, BCOP, BCPS, MSc
Assistant Professor, Department of Pediatrics
Pharmacotherapy for Inherited Metabolic Diseases
Advanced Therapies Department
College of Pharmacy, Experimental and Clinical Pharmacology
University of Minnesota
Minneapolis, MN, United States


Alan Finglas
MSD Action Foundation

Tippi MacKenzie
Professor of Surgery
Division of Pediatric Surgery
UCSF School of Medicine
University of California, San Francisco

Danielle Dong
Rare Diseases Scientific Advocacy Lead, Global Medical Affairs

Heather Park
Head, US Rare Diseases Patient Support Services

Sairei So
Director, International Project Unit
JCR Pharmaceuticals

Heather Lau
Executive Director, Global Clinical Development

Nita Patel
Executive Director, Patient & Professional Advocacy
Amicus Therapeutics

Andres Trevino
Patient Advocacy Director
Chiesi USA, Inc.

Andrea Atherton
Regional Medical Director

Cara O’Neill
Chief Science Officer & Co-Founder
Cure Sanfilippo Foundation

Matthew Ellinwood
Chief Scientific Officer
National MPS Society

Thank you to the sponsors for this session